Youttananukorn T, Konkle B, Peyvandi F, Naccache M, Miesbach W, O’Mahony B, Makris M, Pipe S, Skinner M, Coffin D, Pierce G.Long-term retention plan through myGTR – a patient engagement tool from World Federation of Hemophilia Gene Therapy Registry. (2024), POSTER ABSTRACT (PP-054). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016
Introduction
The World Federation of Haemophilia (WFH) Gene Therapy Registry (GTR) is designed to collect comprehensive clinical data on all people with haemophilia (PWH) who receive gene therapy (GT) globally. To complement data from the GTR, the WFH developed myGTR – a patient engagement tool aimed at collecting patient-reported outcome (PRO) data. PRO data are important and are part of shared decision-making process that could lead to meaningful care and treatment.
Methods
To reduce potential data gap after GT and to ensure long-term engagement between haemophilia treatment centre (HTC) and PWH, the GTR is developing a retention plan. The patient engagement plan of the GTR includes myGTR – the foundational element, data visualisation through dashboards, video/podcasts with experts, and a dedicated website with latest news about GT.
Results
Whilst developing myGTR, the WFH held focus groups to discuss which PRO data are important to collect, at which frequency, and how to capture PWH’s experience on GT. The groups indicated mobile app fatigue and requested a simple tool. myGTR is not available at any app store. It was developed as a web-based application. Patients can choose their preferred contact method – email or text message – to access myGTR and provide PRO data via an interactive digital assis-tant. The PRO data are bleeds, treatments and health related quality of life (HR-QOL) including the Patient Reported Outcomes Burdens and Experiences (PROBE), coreHEM Mental Health Outlook (core-HEM MHO) and EQ-5D-5L. At regular interval (4 times during the first year, 2 times per year thereafter), the patients will be prompted to answer two simple questions about their health status since GT infusion, and complete two out of three HR-QOL questionnaires on a rotational basis.
Conclusions
To monitor GT’s safety, efficacy and long-term effects, engagement from both HTCs and patients is critical. The GTR is a platform for HTCs whereas myGTR allows the patients to continue providing data on their health status and HR-QOL in a simple manner. When working hand-in-hand, both tools can improve patient care and treatment outcomes as well as our understanding of benefits and risks of GT.
Disclosures
Barbara Konkle: Be Biotherapy, Biomarin, Novo Nordisk, Pfizer, Sanofi; Flora Peyvandi: BioMarin Pharmaceutical Inc., CSL Behring, F. Hoffmann-La Roche Ltd., Grifols, Sanofi, Sobi, Takeda; Wolfgang Miesbach: Bayer, BioMarin, Biotest, Chugai, CSL Behring, Free-line, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, Sigilon, Sobi, Takeda/shire; Brian O’Mahony: Biomarin, CSL Behring, Freeline, Irish Haemophilia Society, Pfizer, Roche; Mike Makris: Grifols, Novo Nordisk, Sanofi, Takeda; Steven Pipe: Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Genentech/Roche, GeneVentiv and Equilibra Bioscience, Sanofi, Takeda, Spark Therapeutics, uniQure, Siemens; Mark Skinner: Band Therapeutics, Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Takeda, Vega Therapeutics; Glenn F. Pierce: ASC Therapeutics, Be Bio, BioMarin, Decibel Therapeutics, Frontera, Intellia, Metagenomi, Novo Nordisk, Pfizer, Regeneron, Spark Therapeutics, Third Rock Ventures.
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